Update on the European Medicines Agency recommendation not to renew Translarna marketing authorisation in the European Union

On Friday, 22 September, PTC Therapeutics, the company that manufactures Translarna, issued a statement to Duchenne Patient Advocacy Organisations that provides an update on the European Medicine Agency’s human medicines committee (CHMP) decision not to recommend renewal of the conditional marketing authorization for Translarna.

The statement confirms that PTC will be appealing the decision through an established re-examination process, and that the CHMP opinion does not cover Great Britain (England, Scotland & Wales).  Therefore patients in England, Scotland and Wales are not in scope of the CHMP opinion.

The statement also emphasises that the CHMP did not express concerns regarding the safety of Translarna and that more than 3,000 patients have been treated with Translarna worldwide and more than 700 patients have been involved in the clinical trials.

Under current post-Brexit arrangements, Northern Ireland is within the scope of the CHMP opinion. Patients in Northern Ireland currently receiving Translarna will be able to remain on treatment and new patients will be able to start Translarna during the re-examination process.

According to CHMP guidelines, the re-examination procedure could last until January 2024, with European Commission adoption within 67 days after that i.e. by the end March 2024.

We are monitoring the situation and will continue to keep the community informed of developments.

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